About the Program
A therapeutic approach that uses modified RNA or DNA molecules that bind the UBE3A-ATS (UBE3A antisense transcript). In this program, the ASO is modified to cross the blood brain barrier to enable dosing by IV (intravenous injection) rather than lumbar puncture.
- FAST Pillar
- Pillar 2: Turn on Dad’s UBE3A
- Collaborators
